X4 Pharmaceuticals (XFOR) investor relations material

Strategic focus and operational changes

• Refocused efforts on developing mavorixafor for chronic neutropenia, reducing costs and headcount to align with this goal.

• Deprioritized commercialization of mavorixafor for WHIM syndrome due to limited revenue, but continue to provide the drug to patients in need.

• Shifted medical science liaisons from commercial roles to patient recruitment, leveraging AI and fieldwork to identify and enroll patients in trials.

• Enhanced operational focus on trial enrollment, aiming to complete Phase III enrollment by end of Q3.

Clinical development and trial design

• Mavorixafor is being evaluated in the 4WARD Phase III trial for chronic neutropenia, enrolling 176 patients randomized 1:1 to mavorixafor or placebo, with or without G-CSF.

• Eligibility requires at least two infections in the prior year; primary endpoints are ANC increase and infection reduction.

• Phase I/II data showed mavorixafor increases ANC and can be safely combined with G-CSF, supporting the Phase III design.

• The trial does not allow G-CSF dose titration; a separate Phase II study is planned to address this.

• Long-term safety is monitored, with no new safety signals observed; GI toxicity is managed symptomatically.

Market opportunity and competitive landscape

• Market research estimates 15,000 US patients with symptomatic, severe or moderate chronic neutropenia; initial target is about 5,000 patients.

• Mavorixafor offers an oral alternative to G-CSF, addressing unmet needs such as bone pain, risk of malignancy, and poor tolerability.

• No significant competitors identified in the CXCR4 inhibitor space for this indication.

• Pricing is expected to be premium due to reduced infection rates and rare disease status, though lower than current $500,000 levels.